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Clinical Trial of Pirfenidone in Adult Patients With Neurofibromatosis 1

This study has been completed.
Information provided by:
Mayo Clinic Identifier:
First received: September 16, 2008
Last updated: March 15, 2012
Last verified: March 2012

The study is a phase II, open label trial of oral Pirfenidone in 24 adult patients with neurofibromatosis type 1.

Pirfenidone is a new, broad-spectrum anti-fibrotic drug, with proven in vitro and in vivo negative effects on fibroblast growth and collagen matrix synthesis. Human studies indicate promising therapeutic effects in arresting and reversing fibrosis in a variety of different conditions, where the excessive formation of fibrous tissue is a major pathogenic mechanism. Since the fibrous tissue is a significant component of neurofibroma, reduction of fibrosis could diminish tumor progression and lead to tumor shrinkage. Therefore, Pirfenidone is an excellent candidate for the treatment of plexiform neurofibromas and surgically unresectable tumors in patients with NF1.

Condition Intervention Phase
Drug: Pirfenidone
Phase 2

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: Phase II Clinical Trial of Pirfenidone for the Treatment of Patients With Neurofibromatosis Type I

Resource links provided by NLM:

Further study details as provided by Mayo Clinic:

Primary Outcome Measures:
  • tumor volume [ Time Frame: 24 months ]

Enrollment: 24
Study Start Date: September 2000
Primary Completion Date: August 2004 (Final data collection date for primary outcome measure)
Intervention Details:
    Drug: Pirfenidone
    Capsule 800 mg TID, oral
Detailed Description:

Specific aims of this study are:

  1. To evaluate efficacy of Pirfenidone in NF1 patients with disfiguring or disabling plexiform neurofibroma (PN) and spinal neurofibromas (SN)
  2. To determine the acute, subacute and chronic toxicity of Pirfenidone in patients with NF1.

Ages Eligible for Study:   18 Years to 70 Years   (Adult, Senior)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  • Diagnosis of neurofibromatosis type I, based on clinical criteria (NIH Consensus Development Conference, 1988). Tumors will not be confirmed histologically, since the biopsy could cause a change in tumor growth and such interfere with effect of Pirfenidone treatment.
  • Male or female patients
  • Age 18 years old
  • All patients should be mentally capable of signing the consent form or should have a legal guardian to provide consent
  • Patients who are experiencing symptoms from neurofibromatous lesions and who refuse surgery or are not good surgical candidates, such as those with plexiform neurofibroma who are experiencing significant discomfort, disfigurement or nerve compression or
  • Presence of multiple spinal neurofibromas in which the surgical removal would carry a major risk for spinal cord damage.

Exclusion Criteria:

  • Tumors for which surgical removal could lead to permanent (or long-term) relief of symptoms
  • Patients with open skin lesions and patients for whom surgery is being contemplated or who had surgery less than 4 weeks from starting treatment
  • Patients for whom biopsy is warranted for suspected malignancies
  • Individuals younger than 18 years
  • Pregnant and lactating women
  • Inability to have MR imaging (e.g. claustrophobia, pacemaker or allergy to contrast dye, if administration is needed for neurofibroma imaging)
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its identifier: NCT00754780

Sponsors and Collaborators
Mayo Clinic
Principal Investigator: Dusica Babovic-Vuksanovic, M.D. Mayo Clinic
  More Information

Additional Information:
Responsible Party: Dusica Babovic-Vuksanovic, M.D., Mayo Clinic Identifier: NCT00754780     History of Changes
Other Study ID Numbers: 1835-99
Mayo Pirfenidone Study
Study First Received: September 16, 2008
Last Updated: March 15, 2012

Keywords provided by Mayo Clinic:
plexiform neurofibroma

Additional relevant MeSH terms:
Nerve Sheath Neoplasms
Neoplasms, Nerve Tissue
Neoplasms by Histologic Type
Neoplastic Syndromes, Hereditary
Neurocutaneous Syndromes
Nervous System Diseases
Heredodegenerative Disorders, Nervous System
Neurodegenerative Diseases
Genetic Diseases, Inborn
Peripheral Nervous System Neoplasms
Nervous System Neoplasms
Peripheral Nervous System Diseases
Neuromuscular Diseases
Sensory System Agents
Peripheral Nervous System Agents
Physiological Effects of Drugs
Anti-Inflammatory Agents, Non-Steroidal
Analgesics, Non-Narcotic
Anti-Inflammatory Agents
Antirheumatic Agents
Antineoplastic Agents processed this record on March 28, 2017